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New Gene Therapy Method to help Muscular Dystrophy – Muscle cells rebuilt in Mice Study

December 26th, 2005

New Gene Therapy Method to help Muscular Dystrophy – Muscle cells rebuilt in Mice Study

Stanford University Medical Center

In a press release from Stanford University Medical Center, a new gene therapy technique that was originally intended for skin disease and hemophilia may also have promise for treating muscular dystrophy.  The study conducted at Stanford University School of Medicine, investigated the dystrophin gene in hopes to develop a new treatment for people with muscular dystrophy.

The gene therapy technique is called Rando, named after Thomas Rando MD, PhD an associated professor of neurology and neurological sciences who has been in the process of try to find a cure with gene therapy for muscular dystrophy.  The obstacle has been getting the genes to go into all the muscle cells in the body.  The second challenge is to get the cells to permanently manufacture the beneficial protein created from the gene therapy.

 

Carmen Bertoni, PhD and Michele Calos, PhD and associate professor of genetics developed the Rando gene therapy technique.  The technique offers the potential for a long-term treatment for many genetic diseases, including muscular dystrophy.

Muscular dystrophy is a life shortening and debilitating disease.  The muscle cells break down and are slowly replaced by fat.  People that have muscular dystrophy are usually confined to a wheelchair and die in their 20’s.  At this time there is no cure, but the potential for the use of gene therapy might actually change the course of the disease.

The complete study will be published in the upcoming online edition of the Proceedings of the National Academy of Sciences during the week of Jan. 2nd, 2006.

In the paper Bertoni used a standard gene therapy method to establish dystrophin gene, and another gene that makes a protein glow in mice with muscular dystrophy.  The mice that did not produce enough dystrophin had glowing protein leak out of the cell slowly.  This was a sign that the cell is not healed.  However, when she used Calos’ gene therapy method to establish the same genes, the muscle cells maintained high levels of dystrophin along the length of the cell and the glowing protein did not leak and remained inside the cell.  The thought is that the dystrophin actually repaired the muscle that was deteriorating.

 

"I think our approach has a lot of potential to overcome issues that have slowed the field of gene therapy," Calos said.  Both Calos and Rando mention that the gene therapy cure is still far from being achieved, but Calos is self-assured that her method looks promising and hope it will lead to the cure of hemophilia, skin disease, epidermolysis bullosa as well as muscular dystrophy.

What makes her approach different is that the gene is inserted into the cell’s own DNA, making the correction permanent.  The other benefit to her approach is by not using a virus to disperse the DNA which could have cancer and immune system side effects.  This approach utilized a naked DNA that is able to travel through the bloodstream to the cells.

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By Nicole Wilson
Best Syndication Staff Writer

 

 

Keywords and common misspellings: mucular distrophy musculer dystophy muscler distophy gene jean gen genetic theripy therape


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Copyright 2005 Best Syndication                                            Last Updated Saturday, July 10, 2010 09:46 PM